Results 31 to 40 of about 270,275 (243)

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review

European Journal of Cell Biology, 2023
Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
doaj   +1 more source

Oligonucleotide Formulations Prepared by High-Speed Electrospinning: Maximizing Loading and Exploring Downstream Processability

Pharmaceutics, 2023
The aim of this study was to develop antisense oligonucleotide tablet formulations using high-speed electrospinning. Hydroxypropyl-beta-cyclodextrin (HPβCD) was used as a stabilizer and as an electrospinning matrix. In order to optimize the morphology of
Edit Hirsch, Márió Nacsa, Eszter Pantea, Edina Szabó, Panna Vass, Júlia Domján, Attila Farkas, Zoltán Nyíri, Zsuzsanna Eke, Tamás Vigh, Sune Klint Andersen, Geert Verreck, György János Marosi, Zsombor Kristóf Nagy   +13 more
doaj   +1 more source

Selective Naked-Eye Detection of SARS-CoV-2 Mediated by N Gene Targeted Antisense Oligonucleotide Capped Plasmonic Nanoparticles

ACS Nano, 2020
The current outbreak of the pandemic coronavirus disease 2019 (COVID-19) caused by the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) demands its rapid, convenient, and large-scale diagnosis to downregulate its spread within as well as ...
P. Moitra, M. Alafeef, K. Dighe, M. Frieman, D. Pan   +4 more
semanticscholar   +1 more source

Advances in antisense oligonucleotide development for target identification, validation, and as novel therapeutics [PDF]

, 2008
Antisense oligonucleotides (As-ODNs) are single stranded, synthetically prepared strands of deoxynucleotide sequences, usually 18–21 nucleotides in length, complementary to the mRNA sequence of the target gene.
Alirio J. Melendez, Mansoor, Moizza, Moizza Mansoor, Melendez, Alirio J.   +3 more
core   +1 more source

Identification of differentially expressed sense and antisense transcript pairs in breast epithelial tissues [PDF]

, 2009
Background: More than 20% of human transcripts have naturally occurring antisense products (or natural antisense transcripts – NATs), some of which may play a key role in a range of human diseases.
Oliver, G.R., Kendrick, H., Jat, P., Oliver, Gavin, Neville, A.M., Gavin R Oliver, Howard Kendrick, Tanney, Austin, Tanney, A., Grigoriadis, A., Grigoriadis, Anita, Smalley, M.J., Austin Tanney, Anita Grigoriadis, Kendrick, Howard, Jat, Parmjit, Matt J Smalley, A Munro Neville, Neville, A. Munro, Parmjit Jat, Smalley, Matthew John   +20 more
core   +1 more source

Therapy with 2′-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption

Molecular Therapy: Nucleic Acids, 2019
Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data.
Manoe J. Janssen, Tom T.G. Nieskens, Tessa A.M. Steevels, Pedro Caetano-Pinto, Dirk den Braanker, Melissa Mulder, Yolanda Ponstein, Shaun Jones, Rosalinde Masereeuw, Cathaline den Besten, Martijn J. Wilmer   +10 more
doaj   +1 more source

Preparation of 5'-O-(1-Thiotriphosphate)-modified oligonucleotides using polymerase-endonuclease amplification reaction (PEAR). [PDF]

PLoS ONE, 2013
Antisense oligonucleotides (ASODNs) have been widely used as an important tool for regulating gene expression, and developed into therapeutics. Natural ODNs are susceptible to nuclease degradation, nucleic acid analogues, however, have less side effects,
Biao Li, Shihua Dong, Jiajun Wu, Jianye Zhang, Gang Chen, Quanjiang Dong, Xinhong Zhu, Xiaolong Wang   +7 more
doaj   +1 more source

Taking control of gene expression with light-activated oligonucleotides

BioTechniques, 2007
The recent development of caged oligonucleotides that are efficiently activated by ultraviolet (UV) light creates opportunities for regulating gene expression with very high spatial and temporal resolution.
Ivan J. Dmochowski, XinJing Tang
doaj   +1 more source

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome

Annals of Neurology, 2020
SCN8A encephalopathy is a developmental and epileptic encephalopathy (DEE) caused by de novo gain‐of‐function mutations of sodium channel Nav1.6 that result in neuronal hyperactivity.
Guy M. Lenk, P. Jafar‐nejad, Sophie F. Hill, Lucas D. Huffman, Corrine Smolen, Jacy L. Wagnon, Hayley Petit, Wenxi Yu, Julie Ziobro, K. Bhatia, J. Parent, R. Giger, F. Rigo, M. Meisler   +13 more
semanticscholar   +1 more source

Opportunities and challenges for antisense oligonucleotide therapies

Journal of Inherited Metabolic Disease, 2020
Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that target RNA in a sequence‐specific manner, inducing targeted protein knockdown or restoration. Currently, 10 AON therapies have been approved in the United States
E. Kuijper, A. Bergsma, W. W. M. Pim Pijnappel, Annemieke Aartsma-Rus   +3 more
semanticscholar   +1 more source